Trial · NCT02489344

Evaluation of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-Naïve Adult Male Patients With Fabry Disease

NCT02489344, Phase 2, Completed, Genzyme, a Sanofi Company; results first posted 2019-12-09

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Evaluation of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-Naïve Adult Male Patients With Fabry Disease. Trials, record NCT02489344. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02489344

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Evaluation of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-Naïve Adult Male Patients With Fabry Disease
NCT number: NCT02489344
Status: Completed
Phases: Phase 2
Enrollment: 8 participants, actual
Lead sponsor: Genzyme, a Sanofi Company
Conditions: Fabry Disease
Interventions: GZ/SAR402671
First posted: 2015-07-03
Last updated: 2021-03-23
Other registries' IDs: eudract:2014-004995-49
Results first posted: 2019-12-09

Source ClinicalTrials.gov

Registered outcomes38

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Number of Participants With Treatment Emergent Adverse Events (TEAEs)	From baseline of ACT13739 study up to 37 months post-ACT13739 baseline	Any untoward medical occurrence in a participant who received study drug was considered an adverse event (AE) without regard to possibility of causal relationship with this treatment. TEAEs were defined as AEs that developed or worsened during TEAE period (period from the first administration of study drug in ACT13739 through the last administration of the study drug in the combined ACT13739/LTS14116 treatment period plus 1 month or end of study participation for participant, whichever occurred first). For this analysis, baseline was defined as initial ACT13739 study baseline.
Primary	Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematological Parameters	From baseline of ACT13739 study up to 37 months post-ACT13739 baseline	Criteria for potentially clinically significant abnormalities: * Hemoglobin: less than or equal to (\<=) 115 grams per liter (g/L); greater than or equal to (\>=)185 g/L; decreased from baseline (DFB) \>=20 g/L * Hematocrit: \<=0.37 volume/volume (v/v); \>=0.55 v/v * Erythrocytes: \>=6 Tera/L * Platelets: lesser than (\<) 100 Giga/L; \>=700 Giga/L * Leukocytes: \<3.0 Giga/L (Non-Black \[NB\]) or \<2.0 Giga/L (Black \[B\]); \>=16.0 Giga/L * Neutrophils: \<1.5 Giga/L (NB) or \<1.0 Giga/L (B); * Lymphocytes: greater than (\>) 4.0 Giga/L * Monocytes: \>0.7 Giga/L * Basophils: \>0.1 Giga/L * Eosinophils: \>0.5 Giga/L or \>upper limit of normal (ULN) (if ULN \>=0.5 Giga/L) For this analysis, baseline was defined as initial ACT13739 study baseline.
Primary	Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes	From baseline of ACT13739 study up to 37 months post-ACT13739 baseline	Criteria for potentially clinically significant abnormalities: * Sodium: \<=129 millimoles (mmol)/L; \>=160 mmol/L * Potassium: \<3 mmol/L; \>=5.5 mmol/L * Chloride: \<80 mmol/L; \>115 mmol/L.
Primary	Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters	From baseline of ACT13739 study up to 37 months post-ACT13739 baseline	Criteria for potentially clinically significant abnormalities: * Alanine Aminotransferase (ALT): \>3 ULN; \>5 ULN; \>10 ULN and \>20 ULN * Aspartate aminotransferase (AST): \>3 ULN; \>5 ULN; \>10 ULN and \>20 ULN * Alkaline phosphatase: \>1.5 ULN * Bilirubin: \>1.5 ULN; \>2 ULN.
Primary	Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters	From baseline of ACT13739 study up to 37 months post-ACT13739 baseline	Criteria for potentially clinically significant abnormalities: * Glucose: \<=3.9 mmol/L and \< lower limits of normal (LLN); \>=11.1 mmol/L (unfasted \[unfas\]) or \>=7 mmol/L (fasted \[fas\]) * Lipase: \>= 3 ULN * C Reactive Protein (CRP): \> 2 ULN or \> 10 milligrams (mg)/L (if ULN not provided).
Primary	Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Function Parameters	From baseline of ACT13739 study up to 37 months post-ACT13739 baseline	Criteria for potentially clinically significant abnormalities: * Creatinine: \>=150 micromoles per liter (mcmol/L) (Adults); \>=30% change from baseline; \>= 100% change from baseline * Blood urea nitrogen: \>=17 mmol/L * Urate: \<120 mcmol/L; \>408 mcmol/L For this analysis, baseline was defined as initial ACT13739 study baseline.
Primary	Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Urinalysis	From baseline of ACT13739 study up to 37 months post-ACT13739 baseline	Criteria with potentially clinically significant urine abnormalities: pH: \<= 4.6; pH: \>= 8.0
Primary	Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities	From baseline of ACT13739 study up to 37 months post-ACT13739 baseline	Criteria for potentially clinically significant vital sign abnormalities: * Systolic blood pressure (SBP) supine: \<=95 millimeters of mercury (mmHg) and DFB \>=20 mmHg; \>=160 mmHg and increase from baseline (IFB) \>=20 mmHg * Diastolic blood pressure (DBP) supine: \<=45 mmHg and DFB \>=10 mmHg; \>=110 mmHg and IFB \>=10 mmHg * Heart rate (HR) supine: \<=50 beats per minute (bpm) and DFB \>=20 bpm; \>=120 bpm and IFB \>=20 bpm * Weight: \>=5% DFB; \>=5% IFB For this analysis, baseline was defined as initial ACT13739 study baseline.
Primary	Number of Participants With Potentially Clinically Significant Electrocardiogram (ECG) Abnormalities	From baseline of ACT13739 study up to 37 months post-ACT13739 baseline	Criteria for potentially clinically significant ECG abnormalities: * ECG mean HR: \<30 bpm; \<30 bpm and DFB \>=20 bpm; \<40 bpm; \<40 bpm and DFB \>=20 bpm; \<50 bpm; \<50 bpm and DFB \>=20 bpm; \>90 bpm; \<90 bpm and DFB \>=20 bpm; \>100 bpm; \<100 bpm and DFB \>=20 bpm; \>120 bpm; \<120 bpm and DFB \>=20 bpm * PR Interval: \>200 milliseconds (ms); \>200 ms and IFB \>=25%; \>220 ms; \>220 ms and IFB \>=25%; \>240 ms; \>240 ms and IFB \>=25% * QRS duration: \>110 ms; \>110 ms and IFB \>=25%; \>120 ms; \>120 ms and IFB \>=25% * QTc Bazett (QTcB) interval: \>450 ms; \>480 ms; \>500 ms; IFB \>30 and \<=60 ms, IFB \>60 ms * QTc Fridericia (QTc F): \>450 ms; \>480 ms; \>500 ms; IFB \>30 and \<=60 ms; IFB \>60 ms * QT Interval: \>500 ms For this analysis, baseline was defined as initial ACT13739 study baseline.
Secondary	Change From Baseline in Plasma Globotriaosylceramide (GL-3) Concentration at Weeks 26, 52, 104, and 156	Baseline of ACT13739 study and Weeks 26, 52, 104, and 156 post-ACT13739 baseline	Change from baseline in plasma GL-3 was obtained by subtracting baseline value from post-baseline value at Weeks 26, 52, 104, and 156. Concentration of GL-3 in plasma was determined using a validated liquid chromatography tandem mass spectrometry (LC-MS/MS) method. For this analysis, baseline was defined as initial ACT13739 study baseline.

1 to 10 of 38Source ClinicalTrials.gov

See all 38 registered outcomes

Outcomes38

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Number of Participants With Treatment Emergent Adverse Events (TEAEs)	From baseline of ACT13739 study up to 37 months post-ACT13739 baseline	Analysis was performed on safety population: all participants who received at least 1 dose of investigational medicinal product (IMP) during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis.	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematological Parameters	From baseline of ACT13739 study up to 37 months post-ACT13739 baseline	Analysis was performed on safety population: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis.	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes	From baseline of ACT13739 study up to 37 months post-ACT13739 baseline	Analysis was performed on safety population: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters	From baseline of ACT13739 study up to 37 months post-ACT13739 baseline	Analysis was performed on safety population: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis.	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters	From baseline of ACT13739 study up to 37 months post-ACT13739 baseline	Analysis was performed on safety population: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis.	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Function Parameters	From baseline of ACT13739 study up to 37 months post-ACT13739 baseline	Analysis was performed on safety population: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis.	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Urinalysis	From baseline of ACT13739 study up to 37 months post-ACT13739 baseline	Analysis was performed on safety population: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis.	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities	From baseline of ACT13739 study up to 37 months post-ACT13739 baseline	Analysis was performed on safety population: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis.	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Number of Participants With Potentially Clinically Significant Electrocardiogram (ECG) Abnormalities	From baseline of ACT13739 study up to 37 months post-ACT13739 baseline	Analysis was performed on safety population: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis.	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Change From Baseline in Plasma Globotriaosylceramide (GL-3) Concentration at Weeks 26, 52, 104, and 156	Baseline of ACT13739 study and Weeks 26, 52, 104, and 156 post-ACT13739 baseline	Analysis was performed on full analysis set: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis. Here, 'number analyzed' = participants with available data for each specified category.	micrograms per milliliter (mcg/mL)	MEAN

1 to 10 of 38Source ClinicalTrials.gov

See all 38 outcomes

Results by group453

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Units
Number of Participants With Treatment Emergent Adverse Events (TEAEs)	GZ/SAR402671		9	Participants
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematological Parameters	GZ/SAR402671	Hemoglobin <=115 g/L	1	Participants
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematological Parameters	GZ/SAR402671	Hemoglobin >=185 g/L	0	Participants
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematological Parameters	GZ/SAR402671	Hemoglobin DFB >=20 g/L	0	Participants
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematological Parameters	GZ/SAR402671	Hematocrit <=0.37 v/v	6	Participants
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematological Parameters	GZ/SAR402671	Hematocrit >0.55 v/v	0	Participants
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematological Parameters	GZ/SAR402671	Erythrocytes: >=6 Tera/L	0	Participants
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematological Parameters	GZ/SAR402671	Platelets <100 Giga/L	0	Participants
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematological Parameters	GZ/SAR402671	Platelets >=700 Giga/L	0	Participants
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematological Parameters	GZ/SAR402671	Leukocytes <3.0 Giga/L (NB) or <2.0 Giga/L (B)	0	Participants

1 to 10 of 453Source ClinicalTrials.gov

See all 453 results by group

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
ACT13739 (Initial Study): 26 Weeks	STARTED	GZ/SAR402671	11
ACT13739 (Initial Study): 26 Weeks	COMPLETED	GZ/SAR402671	9
ACT13739 (Initial Study): 26 Weeks	NOT COMPLETED	GZ/SAR402671	2
LTS14116 (Extension Study): 31 Months	STARTED	GZ/SAR402671	8
LTS14116 (Extension Study): 31 Months	COMPLETED	GZ/SAR402671	5
LTS14116 (Extension Study): 31 Months	NOT COMPLETED	GZ/SAR402671	3

Source ClinicalTrials.gov

Adverse events108

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Atrial fibrillation	Cardiac disorders	GZ/SAR402671	1	11
other	Atrioventricular block first degree	Cardiac disorders	GZ/SAR402671	1	11
other	Cardiomyopathy	Cardiac disorders	GZ/SAR402671	2	11
other	Sinus bradycardia	Cardiac disorders	GZ/SAR402671	1	11
other	Deafness unilateral	Ear and labyrinth disorders	GZ/SAR402671	1	11
other	Ear pain	Ear and labyrinth disorders	GZ/SAR402671	1	11
other	Middle ear effusion	Ear and labyrinth disorders	GZ/SAR402671	1	11
other	Tinnitus	Ear and labyrinth disorders	GZ/SAR402671	3	11
other	Vertigo	Ear and labyrinth disorders	GZ/SAR402671	1	11
other	Blepharitis	Eye disorders	GZ/SAR402671	1	11

1 to 10 of 108Source ClinicalTrials.gov

See all 108 adverse events

Adverse event totals3

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	GZ/SAR402671	0	11
Total, other adverse events	GZ/SAR402671	9	11
Total, serious adverse events	GZ/SAR402671	3	11

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
36481125	DERIVED

Source PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

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